Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States

Goshua, George; Calhoun, Cecelia; Ito, Satoko; James, Lyndon; Luviano, Andrea; Krishnamurti, Lakshmanan; Pandya, Ankur

doi:10.7326/m22-3272

Cited by 12 publications

(4 citation statements)

References 24 publications

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“…1 In addition, our conventional analysis with a focus on one group of patients who would benefit from a shared transfusion resource-patients with SCD-does not account for health equity benefits that are present in preventing DHTR-specific mortality. While distributional costeffectiveness analyses for patients with SCD are in their infancy, 14,32 this is a patient population that is both historically marginalized in the United States and one that suffers from a disproportionately high burden of, and genetic predisposition toward, alloimmunization. 33,34 There are several limitations of this study.…”

Section: Discussionmentioning

confidence: 99%

“…The patient population undergoing each strategy is the same and has been previously reported on (Supplement). 10,14 The essential difference between the two strategies is the availability of prior alloantibody data to treating clinicians with strategy #1, abrogating the concern of antibody evanescence and reducing DHTR incidence by some percentage from that currently seen with strategy #2 (i.e., status quo). We test the latter extensively across scenario analyses (see Methods, Scenario Analyses).…”

Section: Simulation Modelmentioning

confidence: 99%

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Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource

Ito,

Pandya,

Hauser

et al. 2024

American J Hematol

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Red blood cell alloimmunization and consequent delayed hemolytic transfusion reaction (DHTR) incidence and mortality in patients with sickle cell disease (SCD) are high. A shared transfusion resource has decreased both in other countries, while in the United States cost concerns persist. We conducted a Markov cohort simulation of a birth cohort of alloimmunized patients with SCD to estimate lifetime DHTR incidence, DHTR‐specific mortality, quality‐adjusted life expectancy (QALE), and costs with the implementation of a shared transfusion resource to identify antibody history versus without (i.e., status quo). We conducted our analysis using a lifetime analytic time horizon and from a United States health system perspective. Implementation of shared transfusion resource projects to decrease cumulative DHTR‐specific mortality by 26% for alloimmunized patients with SCD in the United States, relative to the status quo. For an average patient population of 32 000, this intervention would generate a discounted increment of 4000 QALYs at an incremental discounted cost of $0.3 billion, resulting in an incremental cost‐effectiveness ratio of $75 600/QALY [95% credible interval $70 200–81 400/QALY]. The results are most sensitive to the baseline lifetime medical expenditure of patients with SCD. Alloantibody data exchange is cost‐effective in 100% of 10 000 Monte Carlo simulations. The resource would theoretically need a minimum patient population of 1819 patients or cost no more than $5.29 million annually to be cost‐effective. By reducing DHTR‐specific mortality, a shared transfusion resource in the United States projects to be a life‐saving and cost‐effective intervention for patients with SCD in the United States.

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Section: Discussionmentioning

confidence: 99%

Section: Simulation Modelmentioning

confidence: 99%

Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource

Ito,

Pandya,

Hauser

et al. 2024

American J Hematol

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“…Although cost-effectiveness analysis is an important tool for value-based decision-making, the traditional framework employed by health technology assessment bodies in the US and globally often fails to account for novel value elements (e.g., value of hope, equity) [ 39 ] that may be particularly relevant for one-time therapies with curative intent [ 20 , 21 ]. Accurately assessing the societal value of new therapies is particularly important for SCD, where the potentially transformative clinical benefit of advanced therapies such as lovo-cel may result in far-reaching effects for a patient population and community that remain marginalized [ 26 , 80 ]. Stakeholders and decision-makers are encouraged to consider the broad potential societal benefits of these novel therapies when making value-based decisions related to access and reimbursement.…”

Section: Discussionmentioning

confidence: 99%

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Herring,

Gallagher,

Shah

et al. 2024

PharmacoEconomics

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Background and Objective Gene therapies for sickle cell disease (SCD) may offer meaningful benefits for patients and society. This study evaluated the cost-effectiveness of lovotibeglogene autotemcel (lovo-cel), a one-time gene therapy administered via autologous hematopoietic stem cell transplantation, compared with common care for patients in the United States (US) with SCD aged ≥ 12 years with ≥ 4 vaso-occlusive events (VOEs) in the past 24 months. Methods We developed a patient-level simulation model accounting for lovo-cel and SCD-related events, complications, and mortality over a lifetime time horizon. The pivotal phase 1/2 HGB-206 clinical trial (NCT02140554) served as the basis for lovo-cel efficacy and safety. Cost, quality-of-life, and other clinical data were sourced from HGB-206 data and the literature. Analyses were conducted from US societal and third-party payer perspectives. Uncertainty was assessed through probabilistic sensitivity analysis and extensive scenario analyses. Results Patients treated with lovo-cel were predicted to survive 23.84 years longer on average (standard deviation [SD], 12.80) versus common care (life expectancy, 62.24 versus 38.40 years), with associated discounted patient quality-adjusted life-year (QALY) gains of 10.20 (SD, 4.10) and direct costs avoided of $1,329,201 (SD, $1,346,446) per patient. Predicted societal benefits included discounted caregiver QALY losses avoided of 1.19 (SD, 1.38) and indirect costs avoided of $540,416 (SD, $262,353) per patient. Including lovo-cel costs ($3,282,009 [SD, $29,690] per patient) resulted in incremental cost-effectiveness ratios of $191,519 and $124,051 per QALY gained from third-party payer and societal perspectives, respectively. In scenario analyses, the predicted cost-effectiveness of lovo-cel also was sensitive to baseline age and VOE frequency and to the proportion of patients achieving and maintaining complete resolution of VOEs. Conclusions Our analysis of lovo-cel gene therapy compared with common care for patients in the US with SCD with recurrent VOEs estimated meaningful improvements in survival, quality of life, and other clinical outcomes accompanied by increased overall costs for the health care system and for broader society. The predicted economic value of lovo-cel gene therapy was influenced by uncertainty in long-term clinical effects and by positive spillover effects on patient productivity and caregiver burden. Supplementary Information The online version contains supplementary material available at 10.1007/s40273-024-01385-9.

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“…Bluebird bio set the list price of lovo‐cel at $3.1 million per individual/patient (in line with the pricing of a prior gene therapy to treat beta‐thalassemia; interestingly, the term “equitable access” appears several times in the press release 17 ). Vertex Pharmaceuticals set the list price for exa‐cel at $2.2 million per individual/patient, perhaps reflecting the influence of the cost‐effectiveness analyses, including analyses funded by the NHLBI 18 …”

Section: Sickle Cell Disease Case Studymentioning

confidence: 99%

Translational Research and Health Equity: Gene Therapies for Sickle Cell Disease as a Case Study

Majumder,

Fasipe

2024

Ethics & Human Research

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In August of 2023, the National Academies of Science, Engineering, and Medicine published a timely report titled “Toward Equitable Innovation in Health and Medicine: A Framework.” Here, we review some of the key contributions of the report, focusing on two dimensions of equity: input equity and deployment equity. We then use the example of new gene therapies to treat sickle cell disease (SCD) as a case study of input and deployment equity in translational research. The SCD case study illustrates the need for a kind of translational bioethics with deep understanding of lived experiences and clinical realities as well as a high degree of economic and policy sophistication.

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Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States

Cited by 12 publications

References 24 publications

Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource

Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource

Cost-Effectiveness of Lovotibeglogene Autotemcel (Lovo-Cel) Gene Therapy for Patients with Sickle Cell Disease and Recurrent Vaso-Occlusive Events in the United States

Translational Research and Health Equity: Gene Therapies for Sickle Cell Disease as a Case Study

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